In short
Clinical trials are studying Vutrisiran in people with transthyretin amyloidosis, including those with cardiomyopathy and polyneuropathy. These studies look at safety, efficacy, and heart or nerve-related outcomes in Phase 3 trials. Some trials include people who are continuing treatment, while others compare Vutrisiran with placebo or external control groups.
Key points
- Clinical trials of Vutrisiran are studying people with transthyretin amyloidosis, especially those with cardiomyopathy and those with polyneuropathy. All trials listed here are Phase 3 studies, which means they are late-stage trials with larger groups of patients. The heart studies look at death, hospital visits, urgent heart failure visits, and safety. The nerve studies look at changes in neurologic impairment, including mNIS+7 scores. Some trials are authorised, and one trial has already been completed.
Overview of the clinical research
The trials in this set are studying Vutrisiran in people with transthyretin amyloidosis, a disease where abnormal protein builds up and can harm the heart or nerves. All listed studies are Phase 3 trials, which are later-stage studies designed to test how well a treatment works and how safe it is in larger groups of patients.
Two trials focus on transthyretin amyloidosis with cardiomyopathy, meaning heart muscle disease. Two other studies focus on hereditary transthyretin amyloidosis, including one study of polyneuropathy, which means nerve damage affecting many nerves in the body.
Trials in transthyretin amyloidosis with cardiomyopathy
One authorised Phase 3 trial with NCT ID 2024-518318-25-00 is studying Vutrisiran in patients with transthyretin amyloidosis with cardiomyopathy. The study has 691 planned participants and compares Vutrisiran with 0.9% sodium chloride, which is used here as a placebo-like control.
The main outcome in this trial is a composite outcome, which combines several important events into one result. These events include all-cause mortality, meaning death from any cause, and recurrent cardiovascular events such as hospital stays for heart problems and urgent heart failure visits. The study also looks at a subgroup of patients who were not taking tafamidis at the start of the study, called the Vutrisiran monotherapy subgroup.
A second authorised Phase 3 trial, NCT 2024-518343-38-00, also studies patients with transthyretin amyloidosis with cardiomyopathy. This study plans to enroll 734 patients and focuses on safety, with the main outcome being the frequency of adverse events.
This safety study includes patients who either continued extended use of Vutrisiran or switched from patisiran. That means the trial is looking at safety in people already exposed to related treatment pathways, not only in treatment-naive patients.
Trials in hereditary transthyretin amyloidosis with polyneuropathy
The completed Phase 3 HELIOS-A study, NCT03759379, evaluated Vutrisiran in patients with hereditary transthyretin amyloidosis. It enrolled 135 participants and studied efficacy and safety, with a focus on neurologic impairment.
Its main outcome was the change from baseline in the Modified Neurologic Impairment Score +7, or mNIS+7, compared with the placebo arm of the APOLLO study. Baseline means the starting point before treatment, so this outcome checks whether nerve problems improved or worsened over time.
A separate authorised Phase 3 study, 2025-522544-40-00, is evaluating Vutrisiran in hereditary transthyretin-mediated amyloidosis with polyneuropathy. This trial plans to enroll 186 participants and measures change from baseline in mNIS+7 at Month 9 compared with an external placebo group from the APOLLO study.
This design uses an external control group, which means the comparison group comes from another study rather than from the same trial. The trial is focused on whether Vutrisiran can improve or slow nerve damage in people with hereditary disease.
What the trials measure
The heart trials measure outcomes that matter to patients, such as death, hospitalisation, and urgent visits for heart failure. These outcomes show whether treatment may help people live longer and avoid serious heart-related events.
The safety study measures adverse events, meaning any unwanted medical problems seen during the trial. Safety data are important because they help researchers understand how people respond when treatment is used over time.
The nerve studies measure changes in mNIS+7, a score that tracks neurologic impairment. Lower or improved scores can suggest less nerve damage, while higher scores can suggest worsening disease.
Who may participate
People with ATTR amyloidosis with cardiomyopathy may be eligible for the two heart studies. One of these trials includes patients who are continuing Vutrisiran or who switched from patisiran, so prior treatment history matters in that study.
People with hereditary transthyretin amyloidosis may be eligible for the completed HELIOS-A study, and people with hereditary transthyretin-mediated amyloidosis with polyneuropathy may be eligible for the authorised TRITON-PN study. These trials are aimed at patients whose disease affects the nerves, not only the heart.
Study design and trial phase
All four studies are interventional, which means researchers are giving a treatment and observing what happens. Each one is a Phase 3 trial, showing that the research is in a later stage and is designed to provide stronger evidence about benefit and safety.
Two studies are authorised and ongoing, one study is completed, and one authorised study is focused on nerve disease with a month 9 endpoint. Together, these trials show that Vutrisiran is being studied for both heart-related and nerve-related forms of transthyretin amyloidosis.